- authorisedTherapeutic confirmatory (Phase III)
A Phase 3 randomized, double-blind, placebo-controlled, parallel group, multicenter study with open-label extension to evaluate the efficacy and safety of fenfluramine hydrochloride in study participants with Rett syndrome
This study is looking into a new medicine called Fintepla for people with Rett syndrome. It aims to see if this medicine can help with symptoms and if it's safe to use. Half the participants will get Fintepla, and the other half will get a dummy medicine, to compare the effects.
Spain · Poland · France - active not recruitingPHASE3
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
This study is looking at a new treatment called NGN-401 for girls and women with Rett Syndrome. It aims to see how well and how safely this gene therapy works to help improve symptoms of the condition.
United States · Australia · United Kingdom - active not recruitingPHASE1, PHASE2
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)
This study is looking at a new gene therapy called TSHA-102 for girls aged 5-8 with Rett Syndrome. It aims to see if the treatment is safe and how well it works to help with their condition. It's an early-stage study to learn more about this potential new medicine.
United States · Canada · United Kingdom